This website is for Private Investors* only

*A Private Investor is a recipient of the information who meets all of the conditions set out below, the recipient:

Obtains access to the information in a personal capacity;

Is not required to be regulated or supervised by a body concerned with the regulation or supervision of investment or financial services;

Is not currently registered or qualified as a professional securities trader or investment adviser with any national or state exchange, regulatory authority, professional association or recognised professional body;

Does not currently act in any capacity as an investment adviser, whether or not they have at some time been qualified to do so;

Uses the information solely in relation to the management of their personal funds and not as a trader to the public or for the investment of corporate funds;

Does not distribute, republish or otherwise provide any information or derived works to any third party in any manner or use or process information or derived works for any commercial purposes.

Economy

Market Report

Companies

Currencies

Idorsia begins MODIFY study in patients with Fabry disease

1,691.00

08:09 19/12/18

-2.20%

-38.00

Idorsia announced on Wednesday that the first patient has been enrolled in a registration study to investigate the effect of lucerastat as an oral monotherapy for the treatment of adult patients with genetically confirmed Fabry disease, irrespective of their genetic mutation type.

The company said ‘MODIFY’ would recruit more than 100 patients from 29 trial sites across nine countries.

Its primary endpoint was a reduction in neuropathic pain, described as feeling like burning, shocks or shooting, stabbing, tingling, or pins and needles primarily in the hands and feet.

That major symptom was said to be reported by many patients with Fabry disease as significantly impacting their daily activities and quality of life, despite existing treatment.

“Today's news is an important milestone for the Fabry research and patient communities that have contributed to the development of this study,” said EU coordinating investigator Derralynn Hughes.

“Pain is a genuine and pressing unmet need of the Fabry patient population.”

Dr Hughes said pain remained a “significant burden” for many patients, even for some of those who were already being treated with enzyme replacement therapy.

Idorsia described Fabry disease as a rare, life threatening, inherited lysosomal storage disorder in which a particular lipid called globotriaosylceramide (Gb3) accumulated in cells of many organs of the body.

That build-up resulted in cellular dysfunction leading to a range of signs and symptoms from neuropathic pain, stomach, skin and eye problems, to hypertension, progressive kidney damage, cardiomyopathy, and stroke.

New treatment options were reportedly needed to treat the underlying mechanism of the disease and provide symptomatic relief.